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The Real-Life Drama Behind Clinical Trials

Patients may opt for experimental research when they've exhausted other options.

Darcy Doherty, right, and wife, Rebecca Cumming, in better times in 2009.

Jun 7, 2012

Jeannine Stein, a California native, wrote about health for the Los Angeles Times. In her pursuit of a healthy lifestyle she has taken countless fitness classes, hiked in Nepal and got in a boxing ring.

Clinical trials offer patients a way to get in on the cutting edge of medicine, provided they’re willing to take a gamble on an experimental treatment.

Before new medications, devices, and medical treatments are approved by the Food and Drug Administration, they’re tested via these trials. People volunteer for the studies, which may test the efficacy of a new drug or therapy, such as the benefits of yoga for breast cancer patients. Here are the basics on how people get involved with clinical trials and how they may help:

Who is eligible for a clinical trial?

Each trial has its own criteria for who can participate—some may want men or women, people of a certain age, or patients in various stages of specific diseases. The U.S. National Institutes of Health, which lists clinical trials taking place around the world, reminds potential trial participants that these benchmarks are there to keep people safe, not to exclude them for personal reasons.

Why be part of a clinical trial?

Some people volunteer because they’ve exhausted other treatments, or they want to be on the forefront of new drugs or technology. Not all trials are invasive—some put participants on specific diets or exercise plans to help them shed a few pounds. Although most people sign up for protocols for health reasons, there’s no denying the altruism angle in potentially affecting thousands or millions of people’s lives.

What are the benefits and drawbacks of being part of a trial?

Taking any medication or treatment can be risky—if it works, great, if not, it’s back to square one. Potential side effects, which could include death, are a consideration, as is the possibility of getting a placebo or being put in a control group instead of receiving the drug or treatment one hoped to try. But if the trial results are promising, placebo-takers may get a chance to try the intervention after the trial is over. Note that some trials are only weeks long, while others may last far longer.

Who pays for the trial?

Researchers may have various sponsors, such as pharmaceutical companies, federal agencies, universities and hospitals.

How are patients protected?

Federal regulations protect people in most clinical trials, says the NIH, as are legal and ethical guidelines that oversee other aspects of medicine. Researchers don’t work in a vacuum, but continually report their findings to the trial’s sponsor, as well as government agencies and sometimes at conferences and in medical journals. More checks and balances: The Office for Human Research Protections looks after people who are part of trials being done by the U.S. Department of Health and Human Services. Patients should fully understand their rights and obligations before signing up.

Compassionate use

Clinical trials aren’t the only way patients can get access to experimental medicine. Seriously ill patients who have exhausted other treatments and have not been admitted to trials can request what’s called “compassionate use” of a drug.

Gaining access to investigational drugs can happen two ways, says the American Cancer Society: expanded access programs or single-patient access. Companies that are in late-stage trials with promising medications may allow a group of patients access to treatments through expanded access programs. Individuals can also go through their doctors to ask for the medication. The FDA doesn’t have a problem with this and grants its OK on a case-by-case basis, but the drug companies give the ultimate yea or nay and aren’t legally obligated to work with patients.

One family’s quest for compassionate use of a drug

Going outside of a clinical trial can be a controversial, complicated and emotional process that involves more than just knocking on the door of Big Pharma, as Darcy Doherty has discovered. The 48-year-old from Toronto, Canada, was diagnosed in 2003 in stage IIB melanoma. The former managing director of a financial services company and father of three has gone through various treatments, some of which worked—for a while.

His wife, financial consultant Rebecca Cumming, recently petitioned pharmaceutical company Bristol-Myers Squibb for compassionate use of its medication BMS-936558. “He was getting weaker,” she says.

A recent press release from the pharmaceutical company reported that the antibody showed some success among patients with non small-cell lung cancer, metastatic melanoma and renal cell carcinoma. Doherty tried to get into a clinical trial for the drug earlier this year in Florida, but Cumming said he was denied when tests showed possible evidence of brain tumors. Cumming said her husband responded well in 2007 to the melanoma immunotherapy drug ipilimumab, and she hoped the same would be true of this medication.

So far Bristol-Myers Squibb has denied the request, although she says her dealings with the company have been professional. A story in the Globe and Mail cited a company statement that said the benefits and risks of the drug and patient safety were concerns.

Hitting a wall, Cumming decided to take her husband’s story public, creating a Facebook page and a petition on Change.org to persuade the pharmaceutical company to reconsider her request (to date there are more than 90,000 signatures). So far Bristol-Myers Squibb has not changed its decision. Cumming also recently posted a YouTube video featuring her children and husband, titled, “Help Save Darcy Doherty.”

Video of Help Save Darcy Doherty

“This was a last-ditch attempt, absolutely,” she says. “I felt that maybe the public pressure would work.” While there is no guarantee the medication will help Doherty, without it, Cumming says, her husband will die.

In a similar plea to a pharmaceutical company, Florida breast cancer patient Darlene Gant posted a 20-minute emotional YouTube video in April asking the FDA and pharmaceutical Genentech to grant her compassionate use of the drug pertuzumab, currently up for FDA approval. Genentech initially denied Gant the drug, then relented soon after the video was posted. She is now being treated with it.

While Cumming understands that compassionate requests are typically granted during late-stage trials, and that pharmaceutical companies must make a call on who does and doesn’t get drugs, she still believes more access should be given.

“They’re in the business of saving lives,” she says, “and yet they’re not. They’re saying, sorry, it’s just too risky for you to take that drug. Without it Darcy dies. It’s a risk, but I think it’s the patient’s choice to take that risk.”

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